FDA Approves First Treatment for Certain Patients With Erdheim-Chester Disease

November 6, 2017

ROCKVILLE, Md -- November 6, 2017 -- The US Food and Drug Administration (FDA) expanded the approval of vemurafenib (Zelboraf) to include the treatment of adult patients with Erdheim-Chester Disease (ECD) who have the BRAF V600 mutation

“Today’s approval of [vemurafenib] for patients with ECD demonstrates how we can apply knowledge of the underlying genetic characteristics of certain malignancies to other cancers,” said Richard Pazdur, MD, FDA’s Center for Drug Evaluation and Research, Rockville, Maryland. “This product was first approved in 2011 to treat certain patients with melanoma that harbour the BRAF V600E mutation, and we are now bringing the therapy to patients with a rare cancer with no approved therapies.”

The efficacy of vemurafenib for the treatment of ECD was studied in 22 patients with BRAF-V600-mutation positive ECD. The trial measured the percent of patients who experienced a complete or partial reduction in tumour size (overall response rate). In the trial, 11 patients (50%) experienced a partial response and 1 patient (4.5%) experienced a complete response.

Common side effects of vemurafenib in patients with ECD include arthralgia, maculo-papular rash, alopecia, fatigue, prolonged QT interval, and papilloma.

Severe side effects of vemurafenib include the development of new cancers, growth of tumours in patients with BRAF wild-type melanoma, hypersensitivity reactions (anaphylaxis and DRESS syndrome), severe skin reactions (Stevens-Johnson Syndrome and toxic epidermal necrolysis), heart abnormalities, hepatotoxicity, photosensitivity, uveitis, immune reactions after receiving radiation treatment, renal failure, and Dupuytren’s contracture and plantar fascial fibromatosis.

Vemurafenib can cause harm to a developing fetus; women should be advised of the potential risk to the fetus and to use effective contraception.

The FDA granted this application Priority Review and Breakthrough Therapy designations for this indication. Vemurafenib also received Orphan Drug designation for this indication, which provides incentives to assist and encourage the development of drugs for rare diseases.

SOURCE: US Food and Drug Administration

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